The Neuromuscular Disease Network for Canada


New Publication Guidance on gene replacement therapy in Spinal Muscular Atrophy: a Canadian perspective

The NMD4C is proud to announce that a Guidance on gene replacement therapy in Spinal Muscular Atrophy: a Canadian perspective has been accepted for publication by the Canadian Journal of Neurological Sciences. Notably this consensus statement is authored by NMD4C members Drs. Maryam Oskoui, Hernan Gonorazky, Hugh McMillan, James Dowling, Reshma Amin, Cynthia Gagnon, and Kathryn Selby.

This publication is the first significant research output of the NMD4C, and is a product of the network’s development, dissemination, and implementation of clinical guidelines working group. The working group invited pediatric neuromuscular clinical experts from across the country to provide feedback and direction in adapting existing consensus statements on gene replacement therapy in SMA to the Canadian context.

“It is an exciting time for the neuromuscular field with the advancement of gene therapies. It is our duty as neuromuscular specialists to advocate for safe, equitable, and transparent access to these treatments for all Canadians. We hope that through this consensus we can help inform the application, indication and use of gene replacement therapy in Spinal muscular atrophy.”

– Dr. Hernan Gonorazky.

You can find the publication here.

With three SMA therapies now commercially available in Canada, the working group set out to provide guidance by adapting a set of European consensus statements to a Canadian context and highlighting areas of future need for gene replacement therapy in SMA in Canada. The original publication  from an ad-hoc group of 13 European SMA experts who published a set of consensus statements on the use of gene replacement therapy in SMA using a Delphi consensus process.

The NMD4C will continue to work towards its knowledge translation (KT) goals to improve care standards, train the next generation of neuromuscular researchers and clinicians, and strengthen research infrastructure for better research and more treatments.


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